Challenges in Regulating and Evaluating Precision Medicine Technologies

Precision medicine technologies will likely create challenges for existing regulatory pathways and health technology assessment (HTA) processes and methods. Precision medicine tests, such as whole genome or multigene testing, often fit in a complicated health care pathway for very specific populations, and tend to be based on trials with small sample sizes and limited comparative data. This, along with the increasing volume of tests available and the rapid pace of innovation, makes it difficult to assess their value using traditional HTA methods and processes. Without new and consistent approaches for evaluating new precision medicine technologies, there may be inequalities in who has access to these technologies.

Companion diagnostics and therapeutics often go through separate regulatory and reimbursement pathways. Some regulatory processes are federal and others, such as lab-developed diagnostic tests and diagnostic services, have provincial oversight. Similarly, reimbursement is sometimes at the provincial level, but can also be held by the regional health authority or hospital, depending on the technology and the jurisdiction. These system fragmentations lead to challenges in the funding and reimbursement of precision medicine technologies, and new evaluative approaches will be needed to help balance the need for evaluation and timely access for patients.

Regulatory and HTA processes and methods are adapting by finding new ways of addressing uncertainty and heterogeneity and by harnessing the value of real-world evidence to help inform decision-making. Some examples of work in this area to watch include:

• Generating and using real-world evidence: This can include generating and using real-world evidence through new models of care and health care design. For example, the Canadian Network for Learning Healthcare Systems and Cost-Effective 'Omics Innovation (CLEO) is focused on supporting developing frameworks for learning health systems that enable ongoing evaluations and HTA of precision medicine technologies.
• Exploring new reimbursement and payment models arrangements: Alternative models of payment, such as outcome-based agreements and related approaches, could allow risks to be shared and provide opportunities to change purchasing and funding arrangements based on real-world evidence and ongoing evaluations.

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